The first aim of this thesis was to evaluate the Late Effects Comprehensive Care & Follow-up (LEEF) program after pediatric stem cell transplantation for nonmalignant diseases at the LUMC, and... Show moreThe first aim of this thesis was to evaluate the Late Effects Comprehensive Care & Follow-up (LEEF) program after pediatric stem cell transplantation for nonmalignant diseases at the LUMC, and includes the assessment of various late effects and health-related quality of life. The second aim was to implement and evaluate aspects of VBHC at the LEEF program. This thesis describes lessons learned from the VBHC implementation in the Late Effects Comprehensive Care & Follow-up (LEEF) program and suggests possible future directions for VBHC implementation in similar comprehensive care programs. In this thesis, patient-reported outcomes (PROs) have demonstrated their value in research, such as long-term quality of life studies, but they have also proven to be valuable when used in clinical practice. The evaluation of PROs for both research purposes and clinical use can be expanded within the broader care path of pediatric stem cell transplantation. Furthermore, when discussing long-term HSCT outcomes, it is not solely about survival rates, but PROs are integral to capturing these long-term outcomes. Show less
Rheumatoid arthritis (RA) is a relatively common disease that is characterized by chronic inflammation of joints. The research as described in this thesis focused on the question of whether... Show moreRheumatoid arthritis (RA) is a relatively common disease that is characterized by chronic inflammation of joints. The research as described in this thesis focused on the question of whether adoptive cellular therapy is effective in a mouse model of RA. The most generally known type of adoptive cellular therapy is, probably, bone marrow transplantation (BMT), i.e. BM cells from a healthy donor are transplanted to the patient. Especially, hematological disorders like leukemia are treated with BMT; however, the research presented in this thesis strongly suggests that BMT may also effectively treat diseases, such as RA, in the (near) future. The efficacy of other types of adoptive cellular therapies was also investigated, including the transfer of regulatory T cells. Most strikingly, the disappearance of autoaggressive immune cells was associated with the efficacy of the treatment. These results suggest that these immune cells that, directly or indirectly, cause damage to the patient___s joints were eliminated and/or were kept quiescent. These studies are of importance for the future improvement of the treatment of rheumatic diseases like RA. Show less
